EX-99.2 3 tmb-20230930xex99d2.htm EX-99.2

Exhibit 99.2

ProQR Announces Third Quarter 2023 Operating and Financial Results

Continued advancement platform and initial pipeline programs with liver delivery to address Cholestatic Diseases targeting NTCP and Cardiovascular Disease targeting B4GALT1
Further strengthened leading intellectual property (“IP”) position with issuance of new patent in the United States and successful defense against opposition to its IP in Japan
€120.6 M cash and cash equivalents as of September 30, 2023 providing runway into mid-2026

LEIDEN, Netherlands & CAMBRIDGE, Mass., November 7, 2023 – ProQR Therapeutics NV. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer® RNA editing technology platform, today reported its financial and operating results for the third quarter ended September 30, 2023, and provided a business update.

“We are extremely pleased with the continued progress in advancing our Axiomer RNA editing platform and believe we’re only at the beginning of reaching the potential of this technology,” said Daniel A. de Boer, Chief Executive Officer of ProQR. “We expect that the body of data on our platform and initial pipeline programs will start to ramp up significantly in 2024. Along with our preclinical proof of concept data for the platform, a partnership with Eli Lilly, a leading IP position, and strong cash runway with more than €120.6 million providing runway into mid-2026, we believe ProQR has strong fundamentals in place to execute on our strategy.”

Recent Progress

Continue to advance AX-0810 for patients suffering from cholestatic diseases and AX-1412 for patients with cardiovascular risk. ProQR plans to present and publish additional preclinical data from these two pipeline programs over the next several quarters.
In October, ProQR participated in the Chardan Genetic Medicines Conference panel discussion on “ADAR RNA Editing: Unlocking New Therapeutic Opportunities.”
In November, ProQR announced its IP estate surrounding its Axiomer® RNA editing platform was further strengthened by;
The issuance of a new patent US patent No. 11,781,134 relating to the application of oligonucleotides that are sufficiently complementary to the target sequence to deaminate adenosines using endogenous ADAR, which can deaminate a target adenosine into an inosine in the target sequence. The scope of the new patent further underpins that the broad concept of applying endogenous ADAR by administering antisense oligonucleotides for RNA editing is proprietary to ProQR. Today ProQR has extensive patent protection related to its RNA editing platform Axiomer®, including more than 11 published patent families, that currently comprise a total of 29 patents; and
The Japanese Patent Office issued a final communication indicating that the main claim in one of ProQR’s leading IP estates related to single-stranded antisense RNA editing oligonucleotides (EONs) comprising

one or more mismatches with the target sequence was upheld regardless of a third-party opposition against the granted patent (JP 7074345).

Anticipated Upcoming Events

Present various platform updates over the next several quarters, including liver NHP data, at scientific conferences, as well as research related to ongoing discovery efforts and pipeline programs.
Continue to execute on existing partnership with Eli Lilly and Company (“Lilly”).
ProQR may selectively form new partnerships, which could include multi-target discovery alliances, similar to the Company's partnership with Lilly, or product alliances on specific programs.
ProQR remains on track to advance AX-0810 targeting NTCP and AX-1412 targeting B4GALT1 into clinical development in late 2024/early 2025.

Financial Highlights

On September 30, 2023, ProQR held cash and cash equivalents of €120.6 million, compared to €94.8 million on December 31, 2022. Net cash used in operating activities during the three-month period ended September 30, 2023 was €7.5 million, compared to €15.4 million for the same period last year.

Research and development costs were €5.4 million for the quarter ended September 30, 2023 compared to €15.4 million for the same period last year.

General and administrative costs were €3.3 million for the quarter ended September 30, 2023 compared to €5.4 million for the quarter ended September 30, 2022.

Net loss for the three-month period ended September 30, 2023 was €5.4 million, or €0.07 per diluted share, compared to €23.3 million, or €0.33 per diluted share, for the same period last year. For further financial information for the period ending September 30, 2023, please refer to the financial statements appearing at the end of this release.

About Axiomer®

ProQR is pioneering a next-generation RNA base editing technology called Axiomer®, which could potentially yield a new class of medicines for diverse types of diseases. Axiomer® “Editing Oligonucleotides”, or EONs, mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells called ADAR (Adenosine Deaminase Acting on RNA). Axiomer® EONs are designed to recruit and direct endogenously expressed ADARs to change an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G) – correcting an RNA with a disease-causing mutation back to a normal (wild type) RNA, modulating protein expression, or altering a protein so that it will have a new function that helps prevent or treat disease.

About Biliary Atresia (BA) and Primary Sclerosing Cholangitis (PSC)

Cholestatic disorders refer to a group of diseases presenting excessive and toxic buildup of bile acids in the liver due to bile ducts dysfunction. This leads to liver damage and a range of debilitating symptoms. Without treatment, liver damage can progress through various stages, ultimately leading to liver failure and elevated risk of liver malignancy, affecting life expectancy. Cholestatic diseases remain leading causes of liver transplantation. There are no approved therapies for primary sclerosing cholangitis (PSC) for adults and biliary atresia (BA) for pediatrics. It is estimated that 80,000 and 20,000 individuals have PSC and BA, respectively, in North America and in Europe.


About AX-0810 targeting NTCP

The majority of the bile acids present in the liver cells originate from the enterohepatic reuptake cycle. The key transporter responsible for hepatic uptake of bile acids from portal circulation is the sodium (Na+)-taurocholate cotransporting polypeptide (NTCP, SLC10A1 gene) expressed in the liver. AX-0810 is designed to introduce a loss of function variant in SLC10A1 RNA that has been found in human genetics to prevent re-uptake of bile acids in liver via NTCP. Based on its mechanism of action, AX-0810 has the potential to become a disease modifying treatment for PSC and BA primarily among other cholestatic diseases.

About Cardiovascular Diseases

Cardiovascular diseases (CVDs) are a group of health conditions that affect the heart and blood vessels, such as atherosclerosis which can lead to severe problems like heart attacks, heart failure, and stroke. CVDs represent the leading cause of disability and death in the world. Approximately 18 million people die every year from CVDs representing one third of all the global deaths. Despite available lipid lowering therapies and hypertension medications, the risk of CVDs is still projected to increase rapidly over the coming years.

About AX-1412 targeting B4GALT1

Gene–based analysis of rare beta-1,4-galactosyltransferase 1 (B4GALT1) missense variant (p.Asn352Ser) is known to lead to B4GALT1 protein loss of function and showed an association with decreased coronary artery disease. These beneficial effects are mediated by hypo-galactosylation of the apolipoprotein B100 and fibrinogen, known – independent – drivers of increased risk of CVDs. AX-1412 introduces a protective variant into B4GALT1 RNA to address the remaining residual risk of developing cardiovascular diseases. ProQR intends to advance AX-1412 targeting B4GALT1 to early clinical proof of concept stage, then would seek to partner this program.

About ProQR

ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies. ProQR is pioneering a next-generation RNA technology called Axiomer®, which uses a cell’s own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.

Learn more about ProQR at www.proqr.com.

Forward Looking Statements

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “continue,” "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such forward-looking statements include, but are not limited to, statements regarding our business, preclinical model data, our initial pipeline targets, our Axiomer® platform, our patent estate, including our anticipated strength and our continued investment in it, as well as the timing of our clinical development, the potential of our technologies and product candidates, the collaboration with Lilly and the intended benefits thereof, and our financial position and cash-runway. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. These risks and uncertainties include, among others, the cost, timing and results of preclinical studies and clinical trials and other development activities by us and our collaborative partners whose operations and activities may be slowed or halted shortage and pressure on supply and logistics on the global market; the likelihood of our preclinical and clinical programs being initiated and executed on timelines provided and reliance on our contract research organizations and predictability of timely enrollment of subjects and patients to advance our clinical trials and maintain their own operations; our reliance on contract manufacturers to supply materials for research and development and the risk of supply interruption from a contract


manufacturer; the potential for future data to alter initial and preliminary results of early-stage clinical trials; the unpredictability of the duration and results of the regulatory review of applications or clearances that are necessary to initiate and continue to advance and progress our clinical programs; the ability to secure, maintain and realize the intended benefits of collaborations with partners, including the collaboration with Lilly; the possible impairment of, inability to obtain, and costs to obtain intellectual property rights; possible safety or efficacy concerns that could emerge as new data are generated in research and development; general business, operational, financial and accounting risks, and risks related to litigation and disputes with third parties[; and risks related to macroeconomic conditions and market volatility resulting from global economic developments, geopolitical instability and conflicts]. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law.

ProQR Therapeutics N.V.

Investor contact:
Sarah Kiely
ProQR Therapeutics N.V.
T: +1 617 599 6228
skiely@proqr.com
or

Hans Vitzthum
LifeSci Advisors
T: +1 617 430 7578
hans@lifesciadvisors.com

Media contact:
Robert Stanislaro

FTI Consulting

T: +1 212 850 5657
robert.stanislaro@fticonsulting.com


Financial Tables

PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Financial Position

September 30, 

December 31, 

2023

2022

€1,000

€1,000

Assets

  

  

Current assets

  

  

Cash and cash equivalents

120,552

94,775

Prepayments and other receivables

3,605

59,078

Other taxes

530

607

Total current assets

124,687

154,460

Property, plant and equipment

17,347

16,240

Investments in financial assets

621

Total assets

142,034

171,321

Equity and liabilities

  

  

Equity

  

Equity attributable to owners of the Company

46,340

67,064

Non-controlling interests

(384)

Total equity

46,340

66,680

Current liabilities

  

  

Borrowings

2,344

2,500

Lease liabilities

1,480

1,387

Derivative financial instruments

255

1,263

Trade payables

117

392

Social securities and other taxes

1,230

1,118

Deferred income

16,409

5,641

Other current liabilities

4,814

8,687

Total current liabilities

26,649

20,988

Borrowings

2,891

4,271

Lease liabilities

14,556

13,813

Deferred income

51,598

65,569

Total liabilities

95,694

104,641

Total equity and liabilities

142,034

171,321


PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Profit or Loss and OCI

(€ in thousands, except share and per share data)

Three month period

Nine month period

ended September 30, 

 

ended September 30, 

    

2023

2022

 

2023

2022

€1,000

€1,000

€1,000

€1,000

Revenue

1,370

814

3,230

2,874

Other income

74

80

274

Research and development costs

(5,446)

(15,352)

(17,415)

(40,168)

General and administrative costs

(3,315)

(5,359)

(11,486)

(15,679)

Total operating costs

(8,761)

(20,711)

(28,901)

(55,847)

  

  

  

  

Operating result

(7,391)

(19,823)

(25,591)

(52,699)

Finance income and expense

363

599

289

940

Results related to associates

(8)

Result on derecognition of subsidiary

92

92

Results related to financial liabilities measured at FVTPL

118

5

1,009

3,831

Results on derecognition of financial liabilities

1,357

(4,016)

1,866

(2,872)

  

  

  

  

Result before corporate income taxes

(5,461)

(23,235)

(22,335)

(50,808)

Income taxes

41

(69)

83

(96)

  

  

  

  

Result for the period

(5,420)

(23,304)

(22,252)

(50,904)

Other comprehensive income

Items that will not be reclassified subsequently to profit or loss

Fair value loss on investment in financial asset designated as at FVTOCI

(621)

(621)

Items that may be reclassified subsequently to profit or loss

Foreign exchange differences on translation of foreign operations

286

612

74

1,523

  

  

  

  

Total comprehensive income

(5,755)

(22,692)

(22,799)

(49,381)

Result attributable to

  

  

  

  

Owners of the Company

(5,710)

(23,318)

(22,636)

(51,127)

Non-controlling interests

290

14

384

223

(5,420)

(23,304)

(22,252)

(50,904)

Total comprehensive income attributable to

Owners of the Company

(6,045)

(22,706)

(23,183)

(49,604)

Non-controlling interests

290

14

384

223

(5,755)

(22,692)

(22,799)

(49,381)

Share information

  

  

  

  

Weighted average number of shares outstanding1

81,000,320

71,382,837

80,942,881

71,367,459

Earnings per share attributable to owners of the Company ( per share)

Basic loss per share1

(0.07)

(0.33)

(0.28)

(0.72)

Diluted loss per share1

(0.07)

(0.33)

(0.28)

(0.72)

1.For these periods the potential exercise of share options is not included in the diluted earnings per share as the Company was loss-making. Due to the anti-dilutive nature of the outstanding options, basic and diluted earnings per share are equal.


PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Changes in Equity

Attributable to owners of the Company

  

Number
of shares

  

Share
Capital

  

Share
Premium

  

Equity settled
Employee
Benefit
Reserve

  

Option
premium on
convertible
loan

  

Translation
Reserve

  

Accumulated
Deficit

  

Total

  

Non-
controlling
interests

  

Total
Equity

 

  

€1,000

€1,000

€1,000

€1,000

€1,000

€1,000

€1,000

€1,000

€1,000

Balance at January 1, 2022

 

74,865,381

2,995

398,309

28,443

1,426

430

(316,890)

114,713

(604)

114,109

Result for the period

 

(51,127)

(51,127)

223

(50,904)

Other comprehensive income

 

1,523

1,523

1,523

Recognition of share-based payments

 

3,256

3,256

3,256

Treasury shares transferred

(143,094)

Share options lapsed

(647)

647

Share options exercised

143,094

33

(362)

362

33

33

Balance at September 30, 2022

 

74,865,381

2,995

398,342

30,690

1,426

1,953

(367,008)

68,398

(381)

68,017

Balance at January 1, 2023

 

84,246,967

3,370

412,540

29,052

1,212

(379,110)

67,064

(384)

66,680

Result for the period

 

(22,636)

(22,636)

384

(22,252)

Other comprehensive income

 

74

(621)

(547)

(547)

Recognition of share-based payments

 

2,304

2,304

2,304

Treasury shares transferred

(341,492)

Share options lapsed

(6,209)

6,209

Share options exercised / RSUs vested

341,492

155

(426)

426

155

155

 

  

  

  

  

  

  

  

  

  

  

Balance at September 30, 2023

 

84,246,967

3,370

412,695

24,721

1,286

(395,732)

46,340

46,340


PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Cash Flows

Three month period 

Nine month period

ended September 30, 

 

ended September 30, 

    

2023

2022

2023

2022

€1,000

€1,000

€1,000

€1,000

Cash flows from operating activities

  

  

  

  

Net result

(5,420)

(23,304)

(22,252)

(50,904)

Adjustments for:

— Depreciation

642

612

1,785

1,773

— Share-based compensation

444

1,335

2,304

3,256

— Financial income and expenses

(363)

(599)

(289)

(940)

— Results related to associates

8

— Results related to financial liabilities measured at fair value through profit or loss

(117)

(5)

(1,008)

(3,831)

— Results on derecognition of subsidiary

(131)

(131)

— Results on derecognition of financial liabilities

(1,357)

4,016

(1,866)

2,872

— Income tax

(83)

69

(83)

96

Changes in working capital

(2,008)

3,607

46,660

572

Cash (used in)/generated from operations

(8,393)

(14,269)

25,120

(47,098)

  

  

  

  

Corporate income tax refunds received / (tax paid)

83

(69)

83

(96)

Interest received

802

67

1,667

67

Interest paid

(1,093)

(3,548)

  

  

  

  

Net cash (used in)/generated from operating activities

(7,508)

(15,364)

26,870

(50,675)

  

  

  

  

Cash flow from investing activities

Purchases of property, plant and equipment

(339)

(246)

(769)

(721)

Sales of property, plant and equipment

47

  

  

  

  

Net cash used in investing activities

(339)

(246)

(722)

(721)

  

  

  

  

Cash flow from financing activities

  

  

  

  

Proceeds from exercise of share options

151

155

33

Repayment of convertible loans

(43,373)

(43,373)

Repayment of lease liability

(432)

(381)

(1,338)

(1,314)

  

  

  

  

Net cash used in financing activities

(281)

(43,754)

(1,183)

(44,654)

  

  

  

  

Net (decrease)/increase in cash and cash equivalents

(8,128)

(59,364)

24,965

(96,050)

  

  

  

  

Currency effect cash and cash equivalents

118

3,393

812

8,957

Cash and cash equivalents, at beginning of the period

128,562

156,402

94,775

187,524

  

  

  

  

Cash and cash equivalents at the end of the period

120,552

100,431

120,552

100,431